Goal 2: Reduce Human Disease

Submitted by (@katherinek)

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently ...more »

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Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator1)

Improving blood supply by giving a pig a human bone marrow transplant from a universal donor

What do you think of giving a pig a human bone marrow transplant from a universal donor? The pig could produce blood for human use! Although the pig will eventual die of an autoimmune disease its life can be prolonged with all the new autoimmune medicines we have and/or the pig can be engineered to overexpress PDL1 (or a similar autoimmune suppressor). Additionally even if each pint of blood made this way might be ...more »

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Goal 2: Reduce Human Disease

Submitted by (@andrew.dunham)

Mitigating risks due to the RBC storage lesion and vulnerable patients

What are the underlying dependencies (genomic, metabolic, disease) in individual donors that either accelerate or delay the changes to red blood cells during refrigerated storage? What methods of preparation might protect patients from the risks posed by the accelerated degradation of RBCs provided by "poor storers"? What characteristics of individual patients make them particularly vulnerable to transfusion of red ...more »

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Goal 2: Reduce Human Disease

Submitted by (@nareg.roubinian)

Anemia, oxygen delivery, and red blood cell transfusion

In neonatal, pediatric, and adult patients with critical illness, what is the best means to identify: (1) the degree to which anemia contributes to insufficient oxygen (O2) delivery and (2) the likelihood that O2 delivery will be improved by red blood cell (RBC) transfusion? These questions are most relevant to critically ill populations that exhibit unique physiology, including those with low cardiac output (cardiac ...more »

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Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

Point of care detection of rare cells in blood

Laboratory analyses at the bedside or in the hinterlands can reduce the cost and increase the capture of disease states in underserved populations. The injection of a blood draw directly into a portable device that requires no further operator interventions is a Critical Challenge. With today’s automated chemistry and a miniaturized flow cytometer this challenge could be met.

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Goal 3: Advance Translational Research

Submitted by (@joachim.herz)

Lipid and lipoprotein metabolism in the CNS

The analysis of lipoprotein metabolism has traditionally been restricted to the easily accessible circulation and peripheral tissues. Very little work has been done behind the blood brain barrier, where many of the lipid carrying or metabolizing genes are also expressed. Yet we know very little about their functions there, although for instance ApoE4 is THE primary risk factor for late-onset Alzheimer's disease. Conventional ...more »

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Goal 2: Reduce Human Disease

Submitted by (@katherinek)

Would patients with pulmonary arterial hypertension (PAH) benefit from background anticoagulation in addition to their PAH-targe

Pulmonary hypertension (PH) is a complex, progressive condition characterized by high blood pressure in the lungs. For several decades, oral anticoagulation has been recommended by some societies for patients with a specific form of PH called pulmonary arterial hypertension. However, the evidence currently supporting this recommendation is very limited. To date, no prospective randomized clinical trial has been completed ...more »

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Goal 2: Reduce Human Disease

Submitted by (@nareg.roubinian)

Biology of Red Blood Cell Alloimmunization

What determines which individuals will develop RBC alloimmune responses resulting in clinically meaningful sequelae? This question encompasses: 1) the generation of alloantibodies that limit the availability of compatible blood or cause hemolytic disease of the fetus or newborn (HDFN); 2) the distinction between clinically significant and insignificant alloantibody responses, especially within alloantibody specificities ...more »

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